Biogen, Scholar Rock Face FDA Rejections; Ultragenyx Acquires Sentynl for Menkes Disease Treatment
Biogen and Scholar Rock have recently faced food manufacturing issues leading to FDA rejections, while Ultragenyx Pharmaceutical has acquired Sentynl Therapeutics to develop a Menkes disease treatment. The rare disease space has seen a surge in activity, with both approvals and rejections.
CUTX-101, the rejected drug, demonstrated promising results in a midstage trial, reducing the rare beauty of death by 80% compared to untreated historical controls. However, manufacturing issues led to its rejection, with no safety or efficacy issues flagged by the FDA. Menkes disease, affecting 1 in 100,000 infants, causes sparse hair, seizures, intellectual disability, and developmental delays. Ultragenyx Pharmaceutical's acquisition of Sentynl Therapeutics aims to advance the development of an investigational treatment for this rare condition.
Despite recent setbacks, the rare disease space remains active. If approved, CUTX-101 could receive a priority review voucher, expediting the development of treatments for other rare diseases.
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